Berinert Treatment Proves Effective for Hereditary Angioedema

Data from a study recently published by the Orphanet Journal of Rare Diseases suggests that the self-administration of pnf C1-INH (Berinert) is not only safe, but also that it makes patients more confident in their treatment and improves their quality of life (QoL).

Additionally, the study reports that the self-administered treatment reduces the need for emergency room (ER) admission and help from healthcare professionals for the acute attacks. This, in turn, reduces both overall cost and time between the onset of attacks and treatment.

Hereditary angioedema with C1 inhibitor deficiency is a disabling and potentially fatal condition that is characterized by recurrent episodes of swelling.¹

The observational, monocenter, prospective self-administration of pnf C1-INH in patients with hereditary angiodema (SABHA) study sought to evaluate the safety, tolerability, efficacy, and QoL of patients with hereditary angioedema with C1 inhibitor deficiency with the use of the self-administered injection of pnf C1-INH for IV use.

C1-INH-HAE patients referring to the Luigi Sacco Hospital in Milan composed the study population. Participants from this population were eligible to participate in the study if they were 18 years of age or older and had a clinical and laboratory diagnosis of type I or type II C1-INH-HAE. The median age at enrollment was 42, and 60% of the subjects were female. Patients who participated in the study were required to attend 2 sessions of a self-infusion training course.

Primary data collected in the study included the baseline QoL reported at visit 1, and QoL subsequently reported at visit 2 (month 3) visit 3 (month 6) and visit 4 (month 12), which served as the end of the study, via an HAE-QoL questionnaire. Additionally, the effectiveness, side effects, convenience, and global satisfaction of the drug used were recorded with scores ranging from 1 to 100 for each item (via the Treatment Satisfaction for Medication (TSQM), version 1.4). Characteristics of the attack (duration, severity, and outcome) were also recorded by the patients in personal diaries.

Safety and feasibility of pnf C1-INH self-infusion in a “real-world” setting served as the primary endpoint for the study, and the secondary endpoint was an evaluation of the effect of self-infusion on patients’ QoL, as well as the need for access to the ER after infusion of C1-INH.

According to the results recorded from the 20 enrolled patients, the HAE-QoL total score improved during the study period, with the p value increasing from approximately .49 to .90 over the 12-month study duration.

Positive results were also reported in the TSQM survey, as the effectiveness median score of self-infusion was 67, the median score for the side effects was 100 (extremely positive), and the number of attacks with side effects decreased over time from 37% to 13%. Patient diaries reported very positive results regarding the resolution of symptoms after attacks (97%) and reported that self-infusion was preferred over hospital treatment in more than 80% of cases.

Only 8% of attacks treated with the self-administration of pnf C1-INH required the ER or a healthcare professional (as opposed to the 100% 3 years prior to enrollment).

The study concluded the self-administration of pnf C1-INH as treatment for hereditary angioedema with C1 inhibitor deficiency to be safe, tolerable, effective, and an improvement for the QoL for patients. Since the self-administration of pnf C1-INH also reduced the need of admission to the ER and help from healthcare professionals for acute attacks and the related cost—as well as the time between the onset of attacks and treatments—the self-administration of pnf C1-INH looks like a positive step for those suffering with hereditary angioedema with C1 inhibitor deficiency.

For more data from studies pertaining to the rare disease community, follow Rare Disease Report on Facebook and Twitter.

References:

1. Safety, effectiveness, and impact on quality of life of self-administration with plasma-derived nanofiltered C1 inhibitor (Berinert) in patients with hereditary angioedema: the SABHA study. Orphanet Journal of Rare Diseases. 10, Apr. 2018.